Heart gene therapy trial aims to fix deadly rhythm disorder

NCT ID NCT05885412

Recruiting now Disease control Sponsor: Rocket Pharmaceuticals Inc. Source: ClinicalTrials.gov ↗

First seen Apr 18, 2026 · Last updated May 19, 2026 · Updated 5 times

Summary

This early-stage trial tests a one-time gene therapy for people with a genetic heart condition that causes dangerous heart rhythms. The therapy delivers a working copy of the PKP2 gene to heart cells. The study involves 9 adults with an implanted defibrillator and frequent extra heartbeats. The main goal is to check safety, but researchers will also look for signs that the therapy restores heart protein and reduces abnormal rhythms.

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Contacts and locations

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Study contacts

Contact

Phone: •••-•••-•••• Email: •••••@•••••

Locations

Children's Hospital of Philadelphia
WITHDRAWN

Philadelphia, Pennsylvania, 19104, United States
Duke University
RECRUITING

Durham, North Carolina, 27710, United States

Contact

Contact Email: •••••@•••••
University of California, San Diego
RECRUITING

La Jolla, California, 92037, United States

Contact Phone: •••-•••-•••• Email: •••••@•••••

Conditions

Explore the condition pages connected to this study.

PKP2 ARRHYTHMOGENIC CARDIOMYOPATHY (PKP2-ACM)